Spinocerebellar ataxia type 2: Measures of saccade changes improve power for clinical trials
Mov Disord
; 31(4): 570-8, 2016. tab, graf
Artigo
em Inglês
| CUMED
| ID: cum-76722
Biblioteca responsável:
CU422.1
Localização: CU422.1
ABSTRACT
BACKGROUND:
Saccadic eye movement abnormalities are common in patients with spinocerebellar ataxia type 2, but it is unclear how these alterations progress over time. The aim of this study was to assess the progression of saccade involvement in spinocerebellar ataxia type 2 patients, identify its main determinants, and evaluate its usefulness as outcome measures in clinical trials.METHODS:
A prospective 5-year follow-up study was performed with 30 spinocerebellar ataxia type 2 patients and their matched healthy controls, who were evaluated a total of four times by clinical and electrooculographical assessments of horizontal saccades and by the scoring of ataxia.RESULTS:
Patients showed significant decreases in saccade peak velocity and saccade accuracy as well as increases of saccadic latency during the follow-up period. Annual progression rates were significantly higher in patients compared to controls. Faster progression rates of saccade slowing were associated with higher trinucleotide cytosine-adenine-guanine repeat expansions. Sample-size estimates for two-arm trials would require 19 patients per group to detect a 50 percent reduction in disease progression using saccade peak velocity as outcome variable, but 44 and 124 patients using saccade latency and accuracy, respectively (power, 80 percent; alpha = 0.05).CONCLUSIONS:
Electrooculographical measures of saccade changes are useful for the objective quantification of disease course in spinocerebellar ataxia type 2. The progression rate of saccade slowing is influenced by the expansion size, providing novel insight into the cumulative polyglutamine neurotoxicity, and supporting the usefulness of saccade peak velocity as a sensitive biomarker during the natural history of the disease, and as suitable outcome measure for therapeutic trials (AU)
Texto completo:
Disponível
Coleções:
Bases de dados nacionais
/
Cuba
Base de dados:
CUMED
Assunto principal:
Transtornos da Motilidade Ocular
/
Ataxias Espinocerebelares
Limite:
Adolescente
/
Adulto
/
Idoso
/
Feminino
/
Humanos
/
Masculino
Idioma:
Inglês
Revista:
Mov Disord
Ano de publicação:
2016
Tipo de documento:
Artigo
Instituição/País de afiliação:
Centro para la Investigación y Rehabilitación de las Ataxias Hereditarias/Cuba
/
Centro para la Investigación y Rehabilitación de las Ataxias Hereditarias/Cuba
/
Goethe University Medical School/Alemania
/
University Tübingen/Alemania